The present invention relates to process for the synthesis of novel cationic amphiphilic compounds that facilitate the intracellular delivery of therapeutic biological macromolecules. The present invention also relates to new compositions comprising such cationic amphiphiles those are useful for delivering biological macromolecules into body cells. The novel cationic lipids of the present invention are particularly useful to combat genetic diseases by non-viral gene therapy.
Many defective genes associated with numerous genetic diseases have been identified and characterized. However, since the biological cell membranes are semi-permeable in nature, delivering the required amounts of therapeutically important correct genes into the target body cells is often a daunting challenge. Thus, success of gene therapy approach in treating genetic diseases depends, in a major way, on the development of efficient and safe gene delivery reagents that will facilitate the intracellular delivery of therapeutic genes into the particular body cells of a patient. Accordingly, development of safe and efficient gene delivery reagents and methods that can facilitate entry of functional genes into body cells are of great medical importance.